In the dynamic landscape of pharmaceuticals and healthcare, staying updated on the latest developments in Orphan Drug Designation is of paramount importance. As we delve into the month of August 2023, the world of orphan drugs continues to evolve, bringing with it both exciting breakthroughs and critical alerts. These advancements have the potential to impact the lives of individuals affected by rare diseases profoundly. In this article, we have covered most of the recent news and alerts surrounding Orphan Drug Designation by leading regulatory agencies FDA, EU, and PMDA, among others, shedding light on the innovations, challenges, and regulatory updates that are shaping the future of orphan drug development and accessibility.
List of Orphan Drug Designation August (2023)
| Post Date | Title | Drug/Therapy | Indication/Disease | Key Updates and Announcements | Press Release |
| August 30, 2023 | The Menarini Group Announces ELZONRIS® (Tagraxofusp) Designated as an Orphan Drug for BPDCN by Japanese Ministry of Health, Labor and Welfare | ELZONRIS® (Tagraxofusp) | Blastic Plasmacytoid Dendritic Cell Neoplasm (BPDCN) | In Japan, tagraxofusp is being developed by Nippon Shinyaku, which is currently conducting a Phase 1/2 clinical trial. In March 2021, Stemline and Nippon Shinyaku entered into an exclusive licensing agreement for the development and commercialization of tagraxofusp in Japan, including the possibility of co-promotion. | Orphan Drug Designation for ELZONRIS® (Tagraxofusp) |
| August 29, 2023 | Faron Receives FDA Orphan Drug Designation for Bexmarilimab in Acute Myeloid Leukemia | BEXMAB | Acute Myeloid Leukemia | ODD further strengthens bexmarilimab program by offering clinical development and commercialization benefitsExpected completion of dose escalation, readout of enrichment cohorts, and Phase II initiation in Q4 2023 | Orphan Drug Designation for Bexmarilimab |
| August 23, 2023 | Immix Biopharma — NXC-201 gains Orphan Drug designation in Multiple Myeloma (MM) | NXC-201 | Multiple Myeloma (MM) | With NXC-201, Immix aims to differentiate against currently approved CAR-T therapies that are often associated with neurotoxicity and high-grade cytokine release syndrome (CRS).NXC-201 data appears to provide a competitive safety profile with no serious adverse events reported to date, positioning it as potentially the first outpatient CAR-T therapy. | NXC-201 gains Orphan Drug designation |
| August 21, 2023 | Alligator Bioscience Receives European Medicine Agency Orphan Designation for Mitazalimab in Pancreatic Cancer | Mitazalimab | Pancreatic Cancer | Mitazalimab is a monoclonal antibody targeting CD40 designed to sensitize tumors to chemotherapy and induce immune-mediated tumor killing by activating dendritic cells, B cells, and macrophages.Mitazalimab is currently being evaluated in OPTIMIZE-1, a Phase 2 open-label, multi-center study to assess its safety and efficacy in combination with chemotherapy, mFOLFIRINOX, in previously untreated patients with metastatic pancreatic ductal adenocarcinoma (NCT04888312). | Orphan Designation for Mitazalimab in Pancreatic Cancer |
| August 21, 2023 | Redx Pharma Lung Disease Treatment Receives US FDA Orphan Drug Designation | zelasudil (RXC007) | Idiopathic Pulmonary Fibrosis (IPF). | Zelasudil is an orally available, highly selective small molecule inhibitor that targets ROCK2 which sits at a nodal point in a cell signalling pathway, believed to be central to fibrosis. ROCK2 selectivity is important to avoid systemic hypotension, a serious cardiovascular side effect that has been seen in product candidates that systemically inhibit both ROCK1 and ROCK2. As a selective ROCK2 inhibitor zelasudil, has the potential to treat several fibrotic diseases and has demonstrated robust anti-fibrotic effects in a range of industry-standard in vivo preclinical models, results of which were presented at the International Colloquium on Lung and Airway Fibrosis (ICLAF) and the Antifibrotic Drug Development Summit (AFDD) in 2022. Redx is evaluating zelasudil initially as a treatment for IPF, a severe and life-threatening chronic lung condition with limited treatment options. | Zelasudil granted FDA Orphan Drug Designation |
| August 21, 2023 | CanariaBio Achieves Significant Milestone with FDA’s Orphan Drug Designation for MAb-AR20.5 Targeting Pancreatic Cancer | MAb-AR20.5 | Pancreatic Cancer | MAb-AR20.5 is an investigational drug product. It is an IgG1k type murine monoclonal antibody that binds specifically to the circulating and tumor-associated antigen (MUC1) expressed ubiquitously on pancreatic cancer cells. This milestone marks the first monoclonal antibody targeting Mucin 1 (MUC1) to receive this designation for pancreatic cancer. | FDA’s Orphan Drug Designation for MAb-AR20.5 |
| August 17, 2023 | Nuvectis Pharma Announces Orphan Drug Designation Granted by the FDA to NXP800 for the Treatment of Cholangiocarcinoma | NXP800 | Cholangiocarcinoma | NXP800 is an oral small molecule with a novel mechanism of action that has demonstrated robust activity in several preclinical cancer models, including ARID1a-mutated ovarian, endometrial and gastric carcinomas, as well as cholangiocarcinoma. The clinical activity of NXP800 is currently being evaluated in a Phase 1b clinical trial in patients with platinum resistant, ARID1a-mutated ovarian carcinoma, with additional diseases planned for clinical investigations. | Orphan Drug Designation Granted by the FDA to NXP800 |
| August 15, 2023 | Avidity Biosciences Receives FDA Orphan Drug Designation for AOC 1044 for Treatment of Duchenne Muscular Dystrophy in People with Mutations Amenable to Exon 44 Skipping | AOC 1044 | Duchenne Muscular Dystrophy | AOC 1044 is being assessed in the Phase 1/2 EXPLORE44™ clinical trial for people living with DMD44 and is the first of multiple AOCs in development at Avidity for the treatment of DMD. Avidity plans to share results from the healthy volunteer portion of the EXPLORE44 trial in the fourth quarter of 2023 and is now enrolling participants living with DMD44 into the study.In April 2023, AOC 1044 received FDA Fast Track designation for the treatment of DMD44. | FDA Orphan Drug Designation for AOC 1044 Duchenne Muscular Dystrophy for Treatment |
| August 10, 2023 | Genprex Granted FDA Orphan Drug Designation (ODD) for REQORSA® Immunogene Therapy for the Treatment of Small Cell Lung Cancer | REQORSA® | Small Cell Lung Cancer | In addition to ODD for the treatment of SCLC, in June 2023, the FDA granted Fast Track Designation (FTD) for REQORSA Immunogene Therapy, in combination with Genentech, Inc’s Tecentriq® in patients with extensive-stage small cell lung cancer (ES-SCLC) who did not develop tumor progression after receiving Tecentriq and chemotherapy as initial standard treatment.With ES-SCLC, the cancer has spread from one lung to the other, or to other parts of the body. Extensive-stage is the most common type of SCLC. FDA has also granted Genprex FTD for two other indications of REQORSA Immunogene Therapy, including REQORSA in combination with Tagrisso for non-small cell lung cancer (NSCLC) in patients who have progressed after Tagrisso treatment, and REQORSA in combination with Keytruda for NSCLC in patients who have progressed after Keytruda treatment. | FDA Orphan Drug Designation (ODD) for REQORSA® Immunogene Therapy |
| August 9, 2023 | Bloomsbury Genetic Therapies Receives Orphan Drug Designation from the U.S. FDA for BGT-OTCD for the Treatment of Ornithine Transcarbamylase Deficiency (OTCD) | BGT-OTCD | Ornithine Transcarbamylase Deficiency (OTCD) | BGT-OTCD is an investigational liver-targeted gene therapy for the treatment of OTCD.In May, the UK Medicines and Healthcare products Regulatory Agency (MHRA) approved the clinical trial application submitted by Bloomsbury’s collaborators at University College London (UCL) to initiate a phase 1/2 clinical trial of BGT-OTCD, Halting Ornithine transcarbamylase deficiency with Recombinant AAV in ChildrEn (HORACE), in paediatric patients diagnosed with OTCD, which is expected to begin enrolment in the UK in Q3 2023. | Orphan Drug Designation from the U.S. FDA for BGT-OTCD for the OTCD Treatment |
| August 8, 2023 | Lisata Therapeutics Announces U.S. FDA Orphan Drug Designation Granted to LSTA1 for the Treatment of Malignant Glioma | LSTA1 | Malignant Glioma | LSTA1 is an investigational drug designed to activate a novel uptake pathway that allows co-administered or tethered anti-cancer drugs to penetrate solid tumors more effectively. LSTA1 actuates this active transport system in a tumor-specific manner, resulting in systemically co-administered anti-cancer drugs more efficiently penetrating and accumulating in the tumor. LSTA1 also has the potential to modify the tumor microenvironment, making tumors more susceptible to immunotherapies. Lisata and its collaborators have amassed significant non-clinical data demonstrating enhanced delivery of a range of existing and emerging anti-cancer therapies, including chemotherapeutics, immunotherapies and RNA-based therapeutics. Additionally, LSTA1 has demonstrated favorable safety, tolerability and activity in clinical trials to enhance delivery of standard-of-care chemotherapy for pancreatic cancer. Lisata is exploring the potential of LSTA1 to enable a variety of treatment modalities to treat a range of solid tumors more effectively.Currently, LSTA1 is the subject of multiple ongoing or planned Phase 1b/2a and 2b clinical studies being conducted globally in a variety of solid tumor types in combination with a variety of anti-cancer regimens. In the coming months, the Company, in collaboration with investigators from the University of Tartu in Estonia, plans to initiate a clinical study evaluating LSTA1 in previously untreated glioblastoma multiforme (“GBM”) | FDA Orphan Drug Designation to LSTA1 for the Treatment of Malignant Glioma |
| August 2, 2023 | DTx Pharma Receives FDA Orphan Drug Designation for DTx-1252 for the Treatment of Charcot-Marie-Tooth Disease Type 1A (CMT1A) | DTx-1252 | Charcot-Marie-Tooth Disease Type 1A (CMT1A) | DTx-1252 is a potential first-in-class FALCON™ siRNA therapeutic for treatment of CMT1A. By repressing PMP22, DTx-1252 reverses CMT1A in a mouse model that faithfully recapitulates the genetic and clinical manifestations of the disease. DTx-1252 treatment induces remyelination of axons to normal levels, improves relevant electrophysiological measurements and increases muscle mass, grip strength, coordination and agility in preclinical studies. | FDA Orphan Drug Designation for DTx-1252 |
| August 1, 2023 | Actuate Therapeutics Receives FDA Orphan Drug Designation For Elraglusib For Treatment Of Pancreatic Cancer | Elraglusib (9-ING-41) | Pancreatic Cancer | Elraglusib (9-ING-41) is Actuate’s proprietary small molecule glycogen synthase kinase-3 beta (GSK-3β) inhibitor which is being developed for adults and children with advanced refractory cancers. | Elraglusib (9-ING-41) Get FDA ODD |
| August 1, 2023 | Satellos Bioscience Receives Orphan Drug Designation from the U.S. FDA for SAT-3153 | SAT-3153 | Duchenne Muscular Dystrophy | SAT-3153 is a small molecule designed by Satellos to inhibit a particular kinase protein which the company believes controls Notch polarity within muscle stem cells. Satellos believes this finding explains the progressive muscle destruction which characterizes Duchenne as the muscle tissue cannot repair itself as intended. The Company’s scientists have shown in preclinical studies that inhibiting this kinase protein target enables the modulation of polarity and muscle stem cell divisions, enhances muscle regeneration, and increases muscle mass and critically, muscle function. | SAT-3153 for DMD wins orphan drug, rare pediatric disease status |
| July 31, 2023 | Biohaven’s Taldefgrobep Alfa Receives EU Orphan Drug Designation for Spinal Muscular Atrophy | Taldefgrobep alfa (also known as BHV2000) | Spinal Muscular Atrophy | Taldefgrobep alfa (also known as BHV2000) is a modified adnectin designed to specifically bind to myostatin (GDF-8). Taldefgrobep is a fully human anti-myostatin recombinant protein that lowers free myostatin and acts as an Activin 2b receptor antagonist with the myostatin-taldefgrobep complex. Adnectins are an established proprietary protein therapeutic class based on human fibronectin, an extracellular protein that is naturally abundant in human serum. | EU Orphan Drug Designation to Biohaven’s Taldefgrobep Alfa |
| July 27, 2023 | Akari Therapeutics Granted Nomacopan Orphan Drug Designation from the European Commission for Treatment in Hematopoietic Stem Cell Transplantation | Nomacopan | Hematopoietic Stem Cell Transplantation | Akari’s lead asset, investigational nomacopan, is a bispecific recombinant inhibitor of complement C5 activation and leukotriene B4 (LTB4) activity. Akari is currently conducting a registrational Phase 3 study of nomacopan in pediatric hematopoietic stem cell transplant-related thrombotic microangiopathy (HSCT-TMA). Additionally, the company is moving forward into a Phase 3 double-blind placebo-controlled clinical trial of nomacopan in adult HSCT-TMA with enrollment expected to begin in 2024.Akari has been granted Orphan Drug, Fast Track and Rare Pediatric Disease designations from the FDA for nomacopan for the treatment of pediatric HSCT-TMA. | Orphan Drug Designation to Nomacopan from the European Commission |